Greetings from the ALS Multidisciplinary Clinic in the Sean M. Healey & AMG Center for ALS at Mass General,
Last month’s FDA approval of Qalsody marked a significant milestone in our efforts to solve the challenges of ALS treatments and cures. This therapy dramatically slows the progress of disease in individuals with a specific genetic mutation (SOD1), while also providing important information on the role of neurofilaments for all individuals with ALS. In less than a year, we have seen three new therapies approved by the FDA (oral Radicava, Relyvrio, and Qualsody). In the meantime, our clinical trials continue to grow and basic science researchers are shining new light on critical disease pathways that offer therapeutic targets.
All of our work focuses on improving the care we deliver while we pursue the cures.
Your continued partnership makes our progress possible.
On behalf of all of us at the Healey & AMG Center, thank you!
Merit
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Leading the Charge in ALS Therapy Research and Development
Dr. Cudkowicz and the exciting progress at the Healey & AMG Center is featured this month in Mass General’s Advances in Motion. Dr. Cudkowicz shares her insights on the importance of collaboration and patient-centered care, as well as updates from the clinical trials and encouraging treatments. Read the full article
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FDA grants accelerated approval for ALS treatment Qalsody from Biogen
On April 26, the Federal Drug Administration approved Qalsody, a targeted therapy aimed at slowing the progression of ALS in the subset of people with mutations in the SOD1 gene. Qalsody was tested in the VALOR trial, a double-blind, randomized, placebo-controlled clinical trial, and reduced neurofilament in people with SOD1 ALS. The effectiveness of reducing neurofilament holds promise for developing strategies to reduce it in sporadic ALS as well. The trial was led by Timothy Miller, MD, of Washington University School of Medicine in St. Louis, and Dr. Cudkowicz, Co-Principal Investigator and a critical member of its clinical development since inception.
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Support for ALS Nursing
This month, we celebrated National Nurses Week. Each year we celebrate our nursing colleagues for their dedication to our patients, advocacy for better and safer clinical practice, and, most importantly, their expertise in our divisions. Their dedication to our patients and their families, and the work they do in and out of clinic is pivotal to providing excellent care and high quality research for our patients and families.
We are pleased to share that an anonymous donor has made a multi-year commitment to the Healey & AMG Center for ALS in support of nursing. A portion of the gift will be used to support our nursing team, allowing us to better serve individuals with ALS, providing the personal attention and compassionate care that distinguishes Mass General from other healthcare institutions. The remaining funds will be used to create a grand rounds lecture series that will feature ALS nurse practitioners who are experts in research and care. The science and strategies for ALS care are moving at an ever-increasing pace, and it is critical that our nurses, who remain so close to our patients, have access to the latest information. Philanthropic support is vital to improving access to RNs and funding nursing services not typically covered by insurance. We are grateful to the donor who made this gift possible and all the donors to the Healey & AMG Center for their belief in us. Your partnership is essential to our success.
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EXPANDING ACCESS TO EXPERIMENTAL THERAPIES
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At the Healey & AMG Center, we are dedicated to developing effective treatments for ALS and to providing people with ALS access to experimental therapies through our Expanded Access Protocol (EAP) programs. We continue to partner with several stakeholders to create new EAP models for ALS. Here are the most recent updates: |
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Sean M. Healey & AMG Center for ALS announces first patient dosed in Expanded Access for Trehalose Funded by a grant from the National Institute of Neurological Disorders and Stroke (NINDS)
On April 4, the Healey & AMG Center for ALS at Mass General, in conjunction with Seelos Therapeutics and NINDS, announced the first patient-dosing in the NINDS-funded Expanded Access Protocol for Trehalose. The trehalose ALS EAP allows ALS individuals who are otherwise ineligible for ongoing clinical trials to access this investigational drug. Additionally, this EAP will provide real-world safety, biomarker, and clinical data in ALS individuals treated with long duration of trehalose.
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American Academy of Neurology Annual Meeting 2023
The 2023 American Academy of Neurology (AAN) annual meeting was held April 22- 27 both in person in Boston, MA and virtually. We were proud to have several members of the Healey & AMG Center team present talks, abstracts, posters, and presentations on research and clinical care topics. Highlights included Dr. Cudkowicz delivering an inspiring talk, "Standing on the Shoulders of Giants" that led attendees through the journey of her life and career, as well as Dr. Sabrina Paganoni’s AAN plenary session presentation on the HEALEY Platform Trial. Healey & AMG faculty members like Dr. Suma Babu, Dr. Doreen Ho, Dr. Farrah Mateen, and more also presented during the conference on a range of subjects, from ALS to infectious disease. We are looking forward to the progress we will make over the next year and AAN 2024!
Recordings from the meeting are available here
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ACCELERATING THERAPY DEVELOPMENT |
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The HEALEY ALS Platform Trial continues to make tremendous progress testing multiple investigational drugs. Each drug is tested in a dedicated “regimen” and the trial continues to reach milestones and add new regimens to accelerate the development of effective treatments for ALS. We are excited to share updates for Regimens F & G below: |
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Regimen F, Testing ABBV-CLS-7262
The Healey & AMG Center for ALS, in collaboration with the Northeast ALS Consortium (NEALS), has successfully enrolled its first participants for ABBV-CLS-7262, an investigational product being developed by Calico Life Sciences LLC in collaboration with AbbVie. ABBV-CLS-7262 activates eIF2B, a key regulator of the integrated stress response (ISR), a pathway activated in people with ALS. In neurons exposed to cellular stressors, inhibition of the ISR by ABBV-CLS-7262 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules.
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Regimen G, Testing DNL343
The Healey & AMG Center for ALS, in conjunction with Denali Therapeutics, has announced the first patient-dosing in Regimen G of the HEALEY ALS Platform Trial testing DNL343. DNL343 targets eIF2B, a key regulator of the integrated stress response (ISR). In neurons exposed to cellular stressors, inhibition of the ISR by DNL343 restores protein synthesis and dissolves pre-formed TDP-43 containing stress granules.
DNL343 is being developed by Denali Therapeutics, a publicly owned biotechnology company based in South San Francisco, Calif., and is not yet approved for use in any country.
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Lalji Student Scholar Award
Drs. Shelena and Ayeez Lalji have established the Student Scholar Award in Repair Mechanisms in ALS in honor of Dr. Ayeez Lalji, who was diagnosed in 2017 and is bravely and valiantly living with ALS. Applications for the $5,000 award opened on March 1 and continue through September 1, 2023. Information on eligibility and to apply is available here
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Congratulations to Dr. Tiziana Petrozziello, an instructor in the lab of Dr. Ghazaleh Sadri-Vakili, for receiving a grant from Active Against ALS. This is a 2-year grant, entitled "Determining the functional consequences of the chimeric YAF2-RYBP fusion gene in amyotrophic lateral sclerosis.” |
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CONTRIBUTING TO SCIENTIFIC KNOWLEDGE |
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We continue to contribute to scientific knowledge through research. We are delighted to share that this quarter, we have reported the following:
A full list can be viewed on our website.
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CLINICAL CARE AND COMMUNITY EVENTS |
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The spring has hosted several exciting events for our community and ALS research! |
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ALS Clinic Community and Research Day
On Saturday, May 6, Providers and researchers from the Healey & AMG Center for ALS hosted the annual ALS Community and Clinic Research Day. This served as a chance for people with ALS, families, and other members of our Healey & AMG Center community to hear directly from our faculty, listen to presentations, and have questions answered by the team. Clinicians presented topics such as the HEALEY ALS Platform Trial, EAP program, Neurodegenerative Alzheimer’s Disease and Amyotrophic Lateral Sclerosis (NADALS) trials, as well as an overview of innovation in clinical care. We are grateful to all who attended, and engaged in exciting conversation. We can’t wait to see you again for ALS Clinic Community and Research Day 2024!
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Thanks to the Green Family and the Tackle ALS campaign, launched in September 2018, over $7,000,000 has been raised in support of the HEALEY ALS Platform Trial and the Expanded Access Protocol (EAP) Program at the Sean M. Healey & AMG Center for ALS at Mass General. On April 10th, twenty-two Tackle ALS supporters attended the Buffalo Sabres vs. NY Rangers game after participating in a giveaway for tickets to a box suite donated by NY Rangers defenseman, Adam Fox, a Tackle ALS Team Captain. Proceeds from the giveaway benefit the Tackle ALS campaign which supports the HEALEY ALS Platform Trial. After the game, there was a meet and greet with Adam Fox and the team.
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The Gregg Bonheur ALS Fundraiser was held on April 28th. The night, which included a stand-up comedy routine by Justin Hoff, and live music by Jamie Cain and DJ Soul, was a great success, raising over $100,000 for ALS nursing and research.
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On May 9th, as part of Race to Cure ALS, Jason Goldstein hosted an intimate dinner in New York City to raise funds for the Healey & AMG Center. Dr. Merit Cudkowicz, and nurses, Darlene Sawicki, MSN, NP-BC, and Aimee Roderick, MPH, BSN, RN attended the special evening, with Dr. Cudkowicz providing remarks on the accomplishments and her hope for the future of the Healey & AMG Center. Over $100,000 has been committed so far as a result of the event, and we are expecting even more funds in support of the Center which will go towards the EAP program, the Platform Trial, and funding basic research.
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The Pete Frates 5K Sponsor Thank You and Kickoff was held on May 10th. In November 2022, the annual Pete Frates 5K hosted hundreds of runners from across New England and raised nearly $70,000 to expand the mental-health and patient support program at the ALS multidisciplinary clinic. This year, the race will take place on Sunday, November 5th in Beverly, MA, and funds raised will support the ALS House Call Program. We hope you will consider running with team MGH!
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On May 12th the McCourt Foundation hosted their annual Gala at the Boston Harbor Hotel with over 450 guests in attendance. On June 4th faculty of the Healey & AMG Center, MGH Neurology Department, and their families will get together in Boston’s Seaport for the Foundation’s annual Waterfront 5K. The McCourt Foundation has been supporting research and care at MGH since 2016.
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June 2nd is Lou Gehrig Day!
Healey & AMG Center patient Dick Wolnick and his family will be recognized as part of the pre-game ceremony at Fenway Park. The Healey & AMG Center has been selected as the beneficiary of the MLB’s annual Lou Gehrig Day online auction. Proceeds will support the Healey Center’s Expanded Access Program. will be one of several local charities receiving support from an online auction. The auction will feature autographed baseballs from current and past MLB players as well as gameday experience packages found nowhere else.
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All our work at the Sean M. Healey & AMG Center for ALS combines outstanding, multidisciplinary care with clinical and basic research informed by the needs of our patients. Philanthropy allows us to accelerate the pace of discovery and make a meaningful difference to our patients. We are determined to find the cures. Your partnership will make this possible.
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